News brief­ing: Pfiz­er on track to ear­ly '22 piv­otal read­out on he­mo­phil­ia A; Co­di­ak scales back IPO ex­pec­ta­tions

Pfiz­er and Sang­amo have dosed their first pa­tient in the Phase III gene ther­a­py tri­al of giroc­toco­gene fitel­par­vovec (SB-525) for he­mo­phil­ia A. Re­searchers will be fol­low­ing the ex­pe­ri­ences of pa­tients for 12 months af­ter dose, check­ing on safe­ty, ef­fi­ca­cy and dura­bil­i­ty. The move comes on the heels of a ma­jor set­back at ri­val Bio­Marin, which re­ceived a re­jec­tion let­ter from the FDA, which de­mand­ed more proof of dura­bil­i­ty — a sore point among some of the an­a­lysts track­ing the pro­gram.

A spokesper­son for Pfiz­er tells End­points News to­day that they plan “a piv­otal read­out in ear­ly 2022 for giroc­toco­gene fitel­par­vovec.”

Bren­da Coop­er­stone, Pfiz­er’s chief de­vel­op­ment of­fi­cer, rare dis­ease, not­ed: “En­roll­ment in the lead-in study is pro­gress­ing well and re­cruit­ment is on track for Phase 3. Giv­en the Phase 1/2 study find­ings to date, we be­lieve that giroc­toco­gene fitel­par­vovec has the po­ten­tial to sus­tain fac­tor lev­els and re­duce an­nu­al bleed rates, sug­gest­ing this one-time gene ther­a­py could po­ten­tial­ly trans­form the stan­dard of care for el­i­gi­ble pa­tients world­wide.” — John Car­roll

Co­di­ak scales down IPO ex­pec­ta­tions to $83M

Amid a siz­zling hot biotech IPO en­vi­ron­ment where most com­pa­nies are bring­ing in more than they ini­tial­ly planned, Co­di­ak Bio­Sciences is tun­ing down ex­pec­ta­tions as it sets terms.

Hav­ing pen­ciled in $100 mil­lion in their IPO fil­ing, the ex­o­some spe­cial­ists now say they plan to raise $83 mil­lion by of­fer­ing 5.5 mil­lion shares at a price range of $14 to $16. That’s low­er than what they would have raised in 2019, when they launched but ul­ti­mate­ly pulled an at­tempt to go pub­lic due to un­fa­vor­able mar­ket con­di­tions.

At the mid­point of the pro­posed range, Co­di­ak would com­mand a ful­ly di­lut­ed mar­ket val­ue of $309 mil­lion, ac­cord­ing to Re­nais­sance Cap­i­tal.

A pi­o­neer in co-opt­ing ex­o­somes, the body’s nat­ur­al in­ter­cel­lu­lar com­mu­ni­ca­tion sys­tem, to de­liv­er ther­a­peu­tic pay­loads to spe­cif­ic ad­dress­es, Co­di­ak’s lead pro­gram is de­signed to ac­ti­vate the STING path­way to treat sol­id tu­mors. It ex­pects to start Phase I/II lat­er this year and reap pre­lim­i­nary da­ta by mid-2021. — Am­ber Tong

Pur­due re­port­ed­ly near­ing guilty plea in fed­er­al opi­oid probe

Pur­due Phar­ma is near­ing a plea deal where it would plead guilty to crim­i­nal charges re­lat­ed to its role in ag­gres­sive­ly mar­ket­ing high­ly ad­dic­tive opi­oids, ac­cord­ing to a Reuters re­port.

Run by the Sack­ler fam­i­ly, Pur­due would re­port­ed­ly face fines ex­ceed­ing $8 bil­lion should the agree­ment be reached. Those fines in­clude about $3.54 bil­lion in a crim­i­nal fine, a $2 bil­lion crim­i­nal for­fei­ture and a $2.8 bil­lion civ­il penal­ty, per Reuters

The agree­ment could be reached some­time with­in the next two weeks. Be­cause of Pur­due’s bank­rupt­cy pro­ceed­ings, the fines are un­like­ly to be paid out any­time soon giv­en that the com­pa­ny lacks the cash at present to re­pay all cred­i­tors.

Speak­ing to Reuters, a Jus­tice De­part­ment spokesper­son de­clined to com­ment but said that the re­port “con­tains in­ac­cu­ra­cies and is high­ly mis­lead­ing” with­out ex­plain­ing.

Pros­e­cu­tors across the coun­try, at the fed­er­al and state lev­els, have al­leged Pur­due made bil­lions of dol­lars in prof­its from its opi­oids and sent il­le­gal kick­backs to doc­tors and phar­ma­cies. Pur­due filed for bank­rupt­cy last year fol­low­ing sev­er­al law­suits and crim­i­nal cas­es over its role in fu­el­ing the opi­oid cri­sis. — Max Gel­man

Abeona forms spe­cial com­mit­tee to over­see lead­er­ship

Fol­low­ing a rough few years, Abeona Ther­a­peu­tics an­nounced the for­ma­tion of a spe­cial com­mit­tee of board mem­bers to over­see its ex­ec­u­tive lead­er­ship.

In ad­di­tion, the com­pa­ny has brought in Jef­feries to con­duct a re­view of strate­gic op­er­a­tions. The com­pa­ny said there’s no de­fined time­line for the re­view, and that it doesn’t in­tend to com­ment fur­ther un­less “a spe­cif­ic ini­tia­tive is ap­proved by the board of di­rec­tors, the re­view process is con­clud­ed, or it is oth­er­wise de­ter­mined that oth­er dis­clo­sure is ap­pro­pri­ate.”

Back in 2018, Abeona chief Carsten Thiel was oust­ed fol­low­ing ac­cu­sa­tions of un­spec­i­fied “per­son­al mis­con­duct.” And the fol­low­ing year, the FDA pumped the brakes on the biotech’s Phase III study of its po­ten­tial treat­ment for re­ces­sive dy­s­troph­ic epi­der­mol­y­sis bul­losa. The FDA lift­ed its hold in De­cem­ber, al­low­ing Abeona to re­sume test­ing.

Last Oc­to­ber, Abeona’s $ABEO shares were at $2.38. On Tues­day, they closed at $1.22.

The spe­cial com­mit­tee will work “close­ly with the ex­ec­u­tive lead­er­ship team to de­vel­op the com­pa­ny’s strate­gic di­rec­tion and lead­er­ship plan,” Abeona an­nounced. — Nicole De­Feud­is 

Urovant and Sunovion strike mar­ket­ing deal for po­ten­tial over­ac­tive blad­der drug

Urovant has struck a 5-year deal with Sunovion Phar­ma­ceu­ti­cals to mar­ket its over­ac­tive blad­der drug vibegron in pri­ma­ry care in the US, should it get ap­proval in De­cem­ber.

Walt John­ston

The FDA ac­cept­ed Urovant’s NDA for vibegron in March, and set a PDU­FA date for Dec 26. Urovant in-li­censed the drug from Mer­ck with a $25 mil­lion up­front. Topline Phase III da­ta showed that it hit sta­tis­ti­cal sig­nif­i­cance for both co-pri­ma­ry end­points and all 7 sec­on­daries when com­pared against a place­bo.

Ac­cord­ing to the com­pa­ny, it’s al­so be­ing eval­u­at­ed for OAB in men with be­nign pro­sta­t­ic hy­per­pla­sia, and for ab­dom­i­nal pain as­so­ci­at­ed with ir­ri­ta­ble bow­el syn­drome.

“While Urovant sales rep­re­sen­ta­tives will be fo­cused on urol­o­gists, long-term care, and high-pre­scrib­ing PCPs, the Sunovion team will sig­nif­i­cant­ly broad­en our reach in­to the U.S. pri­ma­ry care com­mu­ni­ty,” Urovant’s se­nior com­mer­cial VP Walt John­ston said in a state­ment.

The new deal is in ad­di­tion to an ex­clu­sive 3-year agree­ment the com­pa­nies reached in June for whole­sale trade and re­tail dis­tri­b­u­tion, con­tract op­er­a­tions and se­lect ac­count man­age­ment for vibegron. — Nicole De­Feud­is 

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Biospec­i­men M&A: Dis­cov­ery ac­quires Al­bert Li's he­pa­to­cyte project; PhI­II tri­al on Bay­er's Nube­qa reached pri­ma­ry end­point

Discovery Life Sciences has acquired what claims to be the Maryland-based host of the world’s largest hepatocyte inventory, known as IVAL, to help researchers select more effective and safer drug candidates in the future.

The combined companies will now serve a wider range of drug research and development scientists, according to Albert Li, who founded IVAL in 2004 and is set to join the Discovery leadership team as the CSO of pharmacology and toxicology.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Re­searchers move clos­er to de­ci­pher­ing blood clots from As­traZeneca, J&J's Covid-19 vac­cines

Researchers may be nearing an answer for the mysterious and life-threatening blood clots that appeared on very rare occasions in people who received the J&J or AstraZeneca Covid-19 vaccine.

The new work builds on an early hypothesis researchers in Norway put forward last spring, when the cases first cropped up. They proposed the events were similar to blood clots that can occur in a small subset of patients who receive heparin, one of the most commonly used blood thinners.